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Profile Name | FGFR1 fusion |
Gene Variant Detail | |
Relevant Treatment Approaches |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
FGFR1 rearrange | myeloproliferative neoplasm | sensitive | Dovitinib | Preclinical | Actionable | In a preclinical study, primary cells from 8p11 myeloproliferative syndrome patients harboring FGFR1 rearrangement were sensitive to Dovitinib (TKI258)-induced growth inhibition in culture (PMID: 17698633). | 17698633 |
FGFR1 mutant | Advanced Solid Tumor | sensitive | Pemigatinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, a variety of cancer cell lines harboring mutations in FGFR1, FGFR2, and/or FGFR3 demonstrated sensitivity to Pemazyre (pemigatinib) in culture and in cell line xenograft models, resulting in inhibition of tumor growth (Cancer Res 2015;75(15 Suppl):Abstract nr 771). | detail... |
FGFR1 rearrange | leukemia | sensitive | Fexagratinib | Preclinical - Cell line xenograft | Actionable | In a preclinical study, AZD4547 inhibited survival of leukemia cells harboring FGFR1 translocation in culture and in cell line xenograft models (PMID: 27550940). | 27550940 |
FGFR1 rearrange | lung small cell carcinoma | sensitive | Fexagratinib | Preclinical - Cell culture | Actionable | In a preclinical study, AZD4547 inhibited survival of small cell lung cancer cells harboring FGFR1 translocation in culture (PMID: 27550940). | 27550940 |
FGFR1 mutant | transitional cell carcinoma | predicted - sensitive | Erdafitinib | Phase II | Actionable | In a Phase II trial, Balversa (erdafitinib) treatment resulted in an objective response rate of 42% (40/96, 3 complete response, 37 partial response) and a disease control rate of 80% in patients with metastatic or unresectable urothelial carcinoma harboring FGFR alterations (J Clin Oncol 36, 2018 (suppl; abstr 4503); NCT02365597). | detail... |
FGFR1 mutant | Advanced Solid Tumor | predicted - sensitive | Zoligratinib | Phase I | Actionable | In a Phase I trial, Debio 1347 treatment resulted in partial response in 10.5% (6/57) and stable disease in 28.1% (16/57) of patients with advanced solid tumors harboring genomic alterations of FGFR1/2/3, including amplifications, fusions, and mutations (PMID: 30745300; NCT01948297). | 30745300 |
FGFR1 rearrange | myeloid and lymphoid neoplasms associated with FGFR1 abnormalities | sensitive | Pemigatinib | Guideline | Actionable | Pemazyre (pemigatinib) is included in guidelines (category 2A) for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring an FGFR1 rearrangement (NCCN.org). | detail... |
FGFR1 rearrange | myeloid and lymphoid neoplasms associated with FGFR1 abnormalities | sensitive | Midostaurin | Guideline | Actionable | Rydapt (midostaurin) is included in guidelines for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring an FGFR1 rearrangement (NCCN.org). | detail... |
FGFR1 rearrange | myeloid and lymphoid neoplasms associated with FGFR1 abnormalities | sensitive | Ponatinib | Guideline | Actionable | Iclusig (ponatinib) is included in guidelines for patients with a myeloid/lymphoid neoplasm with eosinophilia harboring an FGFR1 rearrangement (NCCN.org). | detail... |
FGFR1 rearrange | childhood B-cell acute lymphoblastic leukemia | not applicable | N/A | Guideline | Prognostic | FGFR1 rearrangements are associated with a poor prognosis in pediatric patients with B-cell acute lymphoblastic leukemia (NCCN.org). | detail... |
FGFR1 mutant | Advanced Solid Tumor | predicted - sensitive | ICP-192 | Phase I | Actionable | In a Phase I trial, ICP-192 (gunagratinib) was well-tolerated, and resulted in an overall response rate or 33.3% (4/12, 1 complete response, 3 partial response) and a disease control rate of 91.7% (11/12) in patients with advanced solid tumors harboring FGF/FGFR gene aberrations (J Clin Oncol 39, 2021 (suppl 15; abstr 4092); NCT03758664). | detail... |
FGFR1 rearrange | B-cell acute lymphoblastic leukemia | not applicable | N/A | Guideline | Prognostic | FGFR1 rearrangements are associated with a poor prognosis in patients with B-cell acute lymphoblastic leukemia (NCCN.org). | detail... |
FGFR1 rearrange | myeloid and lymphoid neoplasms associated with FGFR1 abnormalities | sensitive | Pemigatinib | FDA approved | Actionable | In a Phase II trial (FIGHT-203) that supported FDA approval, Pemazyre (pemigatinib) treatment resulted in a complete response rate of 64.5% (20/31) and a complete cytogenetic response rate of 72.2% (24/33) in adult patients with relapsed or refractory myeloid or lymphoid neoplasms harboring FGFR1 rearrangements, with median duration of complete response not reached (Blood (2021) 138 (Supplement 1): 385; NCT03011372). | detail... detail... |
FGFR1 mutant | Advanced Solid Tumor | predicted - sensitive | Erdafitinib | Case Reports/Case Series | Actionable | In a Phase II trial (MATCH), Balversa (erdafitinib) treatment resulted in an objective response rate of 16% (4/25), median progression-free survival of 3.6 months, and median overall survival of 11.0 months in patients with advanced solid tumors harboring FGFR1, FGFR2, or FGFR3 mutations or fusions (PMID: 38603650; NCT02465060). | 38603650 |
FGFR1 rearrange | cholangiocarcinoma | predicted - sensitive | Erdafitinib | Case Reports/Case Series | Actionable | In a Phase II trial, Balversa (erdafitinib) treatment resulted in a confirmed objective response rate (ORR) of 40.9% (9/22, 1 complete, 8 partial responses), a median progression-free survival of 5.6 months, and median overall survival of 25.8 months in patients with cholangiocarcinoma harboring an FGFR rearrangement or FGFR short variant, with an ORR of 57.1% (8/14) with FGFR rearrangement and 12.5% (1/8) with FGFR short variant (PMID: 39138436; NCT02699606). | 39138436 |